Bezuclastinib

Looking to further amp up its immunology business, Sanofi has agreed to acquire Blueprint Medicines in a deal that could be worth as much as $9.5 billion.Blueprints research revolves around an enzyme made by a gene called KIT. This tyrosine kinase enzyme helps control cell development, division and survival, so if it mutates, it can spur the type of uncontrolled cell growth seen in cancer and some rare diseases. Blueprint already has one medicine approved by the Food and Drug Administration, Ayvakit, and is working on a handful of others, two of which are in human testing.The FDA first approved Ayvakit in early 2020 for a rare kind of hard-to-treat gastrointestinal tumor, and specifically for the small subset of adults who have these tumors and certain mutations. Since then, the agency cleared the medicine as a treatment for both the less and more aggressive forms of systemic mastocytosis, an uncommon disorder where a kind of white blood cell known as a mast cell builds up in the bone marrow, digestive tract, skin and other organs. The accumulation can lead to severe inflammation and organ damage.Last year, Blueprint posted $479 million in net product revenue from Ayvakit. The company used to have another marketed cancer drug, Gavreto, which it was co-developing and commercializing with Roche. But Roche ultimately backed away from that alliance, leading Blueprint to sell away rights to Rigel Pharmaceuticals in early 2024.In a statement announcing the deal, Sanofi highlighted the recent trajectory in Ayvakit sales. They were up more than 60%, for example, between the first quarter of 2024 and the same three-month period this year. Blueprint recorded a $67 million net loss in 2024, compared to a more than half-a-billion-dollar net loss in each of the previous two years.The proposed acquisition represents a strategic step forward in our rare and immunology portfolios, Sanofi CEO Paul Hudson said in the Monday statement. It enhances our pipeline and accelerates our transformation into the world's leading immunology company.Hudson added that his company maintains a sizeable capacity for further acquisitions. After being an active dealmaker over the past few years, the French pharmaceutical giant still had close to 8 billion euros in cash and cash equivalents at the end of March.Sanofi bought the diabetes drug developer Provention Bio for almost $3 billion in 2023 and the rare disease-focused Inhibrix for $2.2 billion in 2024. And just last month, it agreed to spend $470 million on Vigil Neuroscience, a Massachusetts-based biotechnology company touting an Alzheimers disease drug that just finished an early-stage human study.Similar to its Vigil proposal, Sanofi is offering Blueprint investors a so-called contingent value right that could be worth up to $6 per Blueprint share, provided one of the biotechs experimental drugs, BLU-808, hits certain development and regulatory goals. BLU-808 is in mid-stage testing as a possible treatment for chronic hives and an allergy-related condition that causes runny eyes and a congested nose. Blueprint also believes the drug could be useful in treating allergic asthma and mast cell activation syndrome.Another Blueprint drug, elenestinib, is further along, having advanced to a late-stage study for the slower-moving form of systemic mastocytosis.Sanofis upfront payment of $9.1 billion values Blueprint shares at $129 apiece, reflecting a premium of about 27% from the biotechs closing stock price on Friday.The transaction is expected to close sometime between July and the end of September, according to Sanofi.Andrew Berens, an analyst at the investment firm Leerink Partners, wrote in a note to clients how his team expected Blueprint to land on the strategic radar of large pharma given Ayvakit is on the path to $1 billion or more in annual sales and the company recently raised its financial guidance for this year. However, the deal came sooner than anticipated, ahead of a key data readout for a rival drug from Cogent Biosciences.Cogents bezuclastinib is also being evaluated as a treatment for systemic mastocytosis, with results set to arrive in the next couple months. Regardless of how strong or weak those results will be, Berens believes theyd serve as a tailwind for Blueprint by removing a key overhang and allowing a strategic acquirer to structure a deal based on assumptions about ... market dynamics.As such, Cogents data could have propped up Blueprints share price, which may have been an impetus for Sanofi to do the deal now, Berens posited. Even so, the analyst still sees Sanofis bid as a positive for Blueprint and this field of research.Cogent shares were down by about 2% late Monday morning. '

Three Registration-Directed Top-line Data Readouts Remain on Track in 2025: SUMMIT in NonAdvanced SM expected in July, APEX in Advanced SM expected in second half of the-year and PEAK in GIST expected by end of year Ended 1Q 2025 with $245.7 million in cash, sufficient to fund operations into late 2026 WALTHAM, Mass. and BOULDER, Colo., May 06, 2025 (GLOBE NEWSWIRE) -- Cogent Biosciences, Inc. (Nasdaq: COGT), a biotechnology company focused on developing precision therapies for genetically defined diseases, today provided a business update and announced financial results for the first quarter ended March 31, 2025. “The first quarter of 2025 was very productive for Cogent as our team focused on executing across our portfolio in preparation for three transformative data readouts this year. We look forward to reporting top-line results from our registration-directed SUMMIT trial with bezuclastinib in patients with nonadvanced systemic mastocytosis in July, followed later in the year with top-line results from our APEX and PEAK trials,” said Andrew Robbins, the Company’s President and Chief Executive Officer. “While we are preparing for a potential launch of bezuclastinib in 2026, we are also very proud of the progress we have made with our early-stage pipeline, including presentations from four distinct programs recently at the annual AACR conference.” Recent Business Highlights Announced expanded clinical results from the Open Label Extension (OLE) portion of the Company’s ongoing SUMMIT trial evaluating bezuclastinib in patients with nonadvanced systemic mastocytosis (NonAdvSM) at the 2025 American Academy of Allergy Asthma & Immunology Annual Meeting (AAAAI) meeting. Bezuclastinib showed a 65% mean improvement in Total Symptom Score (TSS) at 48 weeks, including 88% of patients achieving at least a 50% reduction in TSS. The safety profile for bezuclastinib remains favorable, with adverse events reported primarily as low-grade and reversible. No treatment-related bleeding or cognitive impairment was observed. The most common treatment-related adverse events were hair discoloration and transient elevations in liver transaminases. All cases of elevated transaminases were asymptomatic and fully reversible. Presented updated preclinical data from the company’s KRAS, PI3Kα, FGFR2/3 and ErbB2 candidates at the American Association of Cancer Research (AACR) annual meeting. Bezuclastinib showed a 65% mean improvement in Total Symptom Score (TSS) at 48 weeks, including 88% of patients achieving at least a 50% reduction in TSS. The safety profile for bezuclastinib remains favorable, with adverse events reported primarily as low-grade and reversible. No treatment-related bleeding or cognitive impairment was observed. The most common treatment-related adverse events were hair discoloration and transient elevations in liver transaminases. All cases of elevated transaminases were asymptomatic and fully reversible. Upcoming Milestones Announce top-line results from SUMMIT in July 2025. SUMMIT is a registration-directed, randomized, double-blind, placebo-controlled, global, multicenter, clinical trial of bezuclastinib in patients with NonAdvSM. Announce top-line results from APEX in the second half of 2025. APEX is a registration-directed, global, open-label trial in patients with advanced systemic mastocytosis (AdvSM). Announce top-line results from PEAK by the end of 2025. PEAK is a global, blinded, randomized Phase 3 clinical trial studying the combination of bezuclastinib and sunitinib versus sunitinib alone in patients with imatinib-resistant gastrointestinal stromal tumors (GIST). First Quarter 2025 Financial Results Cash and Cash Equivalents: As of March 31, 2025, cash, cash equivalents and marketable securities were $245.7 million. During the quarter, the Company sold shares under the Company’s at-the-market (ATM) stock offering for net proceeds of $24.3 million and incurred a non-recurring payment of $9.6 million related to annual performance-based bonus compensation. Based on its current plans, the Company expects its existing cash, cash equivalents and marketable securities will be sufficient to fund its operating expenses and capital expenditure requirements into late 2026, including through clinical readouts from ongoing SUMMIT, PEAK, and APEX registration-directed trials. R&D Expenses: Research and development expenses were $63.0 million for the first quarter of 2025 compared to $52.7 million for the first quarter of 2024. The increase was primarily due to costs incurred to support our on-going SUMMIT, PEAK and APEX clinical trials and to the continued progression of our early stage, preclinical and discovery programs. R&D expenses include non-cash stock compensation expense of $5.3 million for the first quarter of 2025 as compared to $4.4 million for the first quarter of 2024. G&A Expenses: General and administrative expenses were $11.9 million for the first quarter of 2025 compared to $9.7 million for the first quarter of 2024. The increase was primarily due to the growth of the organization. G&A expenses include non-cash stock compensation expense of $4.8 million for the first quarter of 2025 as compared to $5.0 million for the first quarter of 2024. Net Loss: Net loss was $72.0 million for the first quarter of 2025 compared to a net loss of $58.3 million for the first quarter of 2024. About Cogent Biosciences, Inc.Cogent Biosciences is a biotechnology company focused on developing precision therapies for genetically defined diseases. The most advanced clinical program, bezuclastinib, is a selective tyrosine kinase inhibitor that is designed to potently inhibit the KIT D816V mutation as well as other mutations in KIT exon 17. KIT D816V is responsible for driving systemic mastocytosis, a serious disease caused by unchecked proliferation of mast cells. Exon 17 mutations are also found in patients with advanced gastrointestinal stromal tumors (GIST), a type of cancer with strong dependence on oncogenic KIT signaling. The company also has an ongoing Phase 1 study of its novel internally discovered FGFR2 inhibitor. In addition, the Cogent Research Team is developing a portfolio of novel targeted therapies to help patients fighting serious, genetically driven diseases targeting mutations in ErbB2, PI3Kα and KRAS. Cogent Biosciences is based in Waltham, MA and Boulder, CO. Visit our website for more information at www.cogentbio.com. Follow Cogent Biosciences on social media: X (formerly known as Twitter) and LinkedIn. Information that may be important to investors will be routinely posted on our website and X. Forward Looking StatementsThis press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, including, but not limited to, statements regarding: the expectation to report SUMMIT top-line results in July 2025; the expectation to report APEX top-line results in the second half of 2025; the expectation to report PEAK top-line results by the end of 2025; the company’s anticipated cash runway into late 2026; and the potential commercial launch of bezuclastinib in 2026. The use of words such as, but not limited to, "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "might," "plan," "potential," "predict," "project," "should," "target," "will," or "would" and similar words expressions are intended to identify forward-looking statements. Forward-looking statements are neither historical facts nor assurances of future performance. Instead, they are based on our current beliefs, expectations and assumptions regarding the future of our business, future plans and strategies, our clinical results, the rate of enrollment in our clinical trials and other future conditions. New risks and uncertainties may emerge from time to time, and it is not possible to predict all risks and uncertainties. No representations or warranties (expressed or implied) are made about the accuracy of any such forward-looking statements. We may not actually achieve the forecasts or milestones disclosed in our forward-looking statements, and you should not place undue reliance on our forward-looking statements. Such forward-looking statements are subject to a number of material risks and uncertainties including but not limited to those set forth under the caption "Risk Factors" in Cogent's most recent Annual Report on Form 10-K filed with the SEC, as well as discussions of potential risks, uncertainties, and other important factors in our subsequent filings with the SEC. Any forward-looking statement speaks only as of the date on which it was made. Neither we, nor our affiliates, advisors or representatives, undertake any obligation to publicly update or revise any forward-looking statement, whether as result of new information, future events or otherwise, except as required by law. These forward-looking statements should not be relied upon as representing our views as of any date subsequent to the date hereof. Contact:Christi WaarichSenior Director, Investor Relationschristi.waarich@cogentbio.com617-830-1653